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GVHDPhase 1NCT04473911

Haplo Peripheral Blood Sct In GVHD Prevention

A Phase 1 study testing FLUDARABINE, CYCLOPHOSPHAMIDE and TBI in people with GVHD and AML, run by Zachariah Michael DeFilipp at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This research study is studying the RGI-2001 for preventing Graft-vs-Host Disease (GVHD) in people with acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), myelodysplastic syndrome (MDS), myeloproliferative disorders (MPN), chronic myelomonocytic leukemic (CMML), chemosensitive hodgkin lymphoma (HL), or… Read more about this study

Who is it for?
Ages 18 to 80 years
Conditions the study is for
GVHD, AML, ALL
What is being tested?
FLUDARABINE, CYCLOPHOSPHAMIDE and TBI
Could I receive a placebo?
No, everyone receives an active treatment
How many people
25 participants
Where
1 siteUnited States
Study dates
14 Aug 2020to 19 Aug 2024
Phase
Phase 1
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 80 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Men or women ≥ 18 and ≤ 80 years old
  • Diagnosis of hematological malignancy:
  • Acute myeloid leukemia (AML) or acute lymphoblastic leukemia (ALL) in morphologic complete remission
  • Myelodysplastic syndrome (MDS), myeloproliferative disorders (MPN), or chronic myelomonocytic leukemic (CMML) with < 5% blasts in blood or bone marrow
  • Chemosensitive Hodgkin lymphoma (HL) or Non-Hodgkin lymphoma (NHL)
  • Patients must be undergoing haploidentical allogeneic hematopoietic cell transplantation, defined as 1st or 2nd degree relative with at least 5/10 matching at HLA-A, -B, -C, DR, and DQ.
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  • ECOG performance status ≤2
  • Patients with adequate physical function as measured by:
  • Cardiac: Left ventricular ejection fraction at rest must be ≥ 40%, or shortening fraction >25%
  • Hepatic:
  • Bilirubin ≤ 2.5 mg/dL, except for patients with Gilbert's syndrome or hemolysis
  • ALT, AST, and Alkaline Phosphatase < 5 x ULN
  • Renal: Serum creatinine within normal range, or if serum creatinine is outside normal range, then renal function (measured or estimated creatinine clearance or GFR) ≥ 40mL/min/1.73m2
  • Pulmonary: DLCO (corrected for hemoglobin), FEV1 and FVC ≥ 50% predicted
  • Ability to understand and the willingness to sign a written informed consent document

Who cannot join

  • Prior allogeneic hematopoietic stem cell transplantation. (Patients may have received a prior autologous hematopoietic stem cell transplant.)
  • Participants who are receiving any other investigational agents within 14 days prior to RGI-2001 dosing. Thus, participants must stop investigational agents by Day -9 prior to transplant.
  • Uncontrolled intercurrent illness including, but not limited to, symptomatic congestive heart failure, recent myocardial infarction or stroke, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
  • Patients with active or uncontrolled bacterial, viral, or fungal infection(s) requiring systemic therapy.
  • Planned use of prophylactic donor lymphocyte infusion (DLI) therapy.
  • Pregnant and breast-feeding women are ineligible because they are not eligible for hematopoietic stem cell transplantation.
  • HIV-positive participants and patients with active Hepatitis B or C are ineligible

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Massachusetts General Hospital

    Boston, Massachusetts, 02115

Other trials in United States

About this study

Official title: Reduced Intensity Haploidentical Peripheral Blood Stem Cell Transplantation With Post-transplant Cyclophosphamide and Sirolimus/Mycophenolate Mofetil/RGI-2001 Based GVHD Prevention: a Pilot Study

This research study is studying the RGI-2001 for preventing Graft-vs-Host Disease (GVHD) in people with acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), myelodysplastic syndrome (MDS), myeloproliferative disorders (MPN), chronic myelomonocytic leukemic (CMML), chemosensitive hodgkin lymphoma (HL), or Non-Hodgkin lymphoma (NHL).who will have a blood stem cell transplantation.

  • GVHD is a condition in which cells from the donor's tissue attack the organs.
  • RGI-2001 is an investigational treatment
Read the detailed description
  • This is a pilot study in subjects undergoing reduced-intensity haploidentical peripheral blood stem cell transplantation who will receive graft-versus-host disease prevention with post-transplant cyclophosphamide, followed by sirolimus, mycophenolate mofetil, and RGI-2001.
  • The research study procedures include screening for eligibility and study treatment including evaluations and follow up visits.
  • The standard of care drugs of fludarabine, cyclophosphamide, melphalan, radiation, sirolimus, and mycophenolate mofetil are all FDA approved.
  • Eligible Participants will be placed in 1 of 2 groups, per physicians discretion:
  • Regimen #1 :
  • Before stem cell transplant:Fludarabine + Cyclophosphamide + Radiation
  • After stem cell transplant: Cyclophosphamide + Sirolimus +Mycophenolate mofetil + RGI-2001
  • Regimen #2
  • Before stem cell transplant: fludarabine + melphalan + radiation
  • After stem cell transplant: cyclophosphamide + sirolimus +Mycophenolate mofetil + RGI-2001
  • A total of 20 participants will be enrolled to this trial
  • The U.S. Food and Drug Administration (FDA) has not approved RGI-2001 as a treatment for any disease.
Conditions
GVHD, AML, ALL, MDS, MPN, CMML
Keywords
GVHD, AML, ALL, MDS, MPN, CMML, Hodgkin Lymphoma, Non Hodgkin Lymphoma, Blood Stem Cell Transplant Failure, Graft Vs Host Disease, Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Myelodysplastic Syndromes, Myeloproliferative Disorder, Chronic Myelomonocytic Leukemia, Chemosensitive Hodgkin Lymphoma
Medical subject headings
Hodgkin Disease, Lymphoma, Non-Hodgkin, Graft vs Host Disease, Leukemia, Myeloid, Acute, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Myelodysplastic Syndromes, Myeloproliferative Disorders, Leukemia, Myelomonocytic, Chronic

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • FLUDARABINEDrug

    Also called Fludara®

    predetermined dose, intravenously, a predetermined times per cycle Given in both pre stem cell and post stem cell cycles

  • CYCLOPHOSPHAMIDEDrug

    Also called Cytoxan®, Neosar®

    ◦Cyclophosphamide predetermined dose, predetermined number of times in Given in pre-stem cell Regimen #1 Cyclophosphamide predetermined dose, predetermined number of times in cycle, intravenous infusion

  • TBIRadiation

    Total body irradiation (TBI) once per cycle.

  • MelphalanDrug

    Also called Alkeran®, L-PAM, L-Sarcolysin, Phenylalanine Mustard

    Melphalan, infusion, determined dosage, once per cycle

  • SirolimusDrug

    Also called Rapamune

    Sirolimus: Predetermined dosage, predetermined number of time in cycle, oral: Please note that doses of sirolimus can be adjusted at the treating physician's discretion given the multiple drugs and other situations which affect its metabolism

  • Mycophenolate mofetilDrug

    Also called CellCept, Myfortic

    ◦Mycophenolate mofetil, oral or iv(predetermined dose or IV TID (based upon actual body weight), at predetermined times per cycle

  • RGI-2001Drug

    IV, predetermined dose, weekly to 6 total doses

  • CYCLOPHOSPHAMIDEDrug

    Also called Cytoxan®, Neosar®

    ◦Given in Post Stem Cell cycle of Regimen #1 and #2 Cyclophosphamide predetermined dose, predetermined number of times in cycle, intravenous infusion

Study groups in NCT04473911
Study groupRoleWhat they receive
Regimen 1: Fludarabine, Cyclophosphamide, and TBIExperimental-. Patients who meet eligibility criteria for the study will subsequently be enrolled for treatment. Two reduced intensity regimens will be allowed, according to the choice of the treating physician * Pre- stem cell transplant: * Fludarabine predetermined dose, intravenously, 4 times per cycle * Cyclophosphamide predetermined dose, predetermined number of times in cycle, intravenous infusion * Total body irradiation (TBI) once during treatment cycle * Post stem cell transplant: * Cyclophosphamide predetermined dose, predetermined number of times in cycle, intravenous infusion * Sirolimus: Predetermined dosage, predetermined number of time in cycle, oral. * Mycophenolate mofetil, oral or iv(predetermined dose or IV TID (based upon actual body weight), at predetermined times per cycle * RGI-2001: IV, predetermined dose, weekly to 6 total doses
Receives: Drug: FLUDARABINE; Drug: CYCLOPHOSPHAMIDE; Radiation: TBI; Drug: Sirolimus; Drug: Mycophenolate mofetil; Drug: RGI-2001
Regimen 2: Fludarabine, Melphalan, and TBIExperimental-. Patients who meet eligibility criteria for the study will subsequently be enrolled for treatment. Two reduced intensity regimens will be allowed, according to the choice of the treating physician * Pre- stem cell transplant: * Fludarabine predetermined dose, intravenously 3 times per cycle * Melphalan, infusion, determined dosage, once per cycle * Total body irradiation (TBI) once per cycle. * Post stem cell transplant * Cyclophosphamide predetermined dose, predetermined number of times in cycle, intravenous infusion * Sirolimus: Predetermined dosage, predetermined number of time in cycle, oral: * Mycophenolate mofetil, oral or iv(predetermined dose or IV TID (based upon actual body weight), at predetermined times per cycle * RGI-2001: IV, predetermined dose, weekly to 6 total doses
Receives: Drug: FLUDARABINE; Radiation: TBI; Drug: Melphalan; Drug: Sirolimus; Drug: Mycophenolate mofetil; Drug: RGI-2001; Drug: CYCLOPHOSPHAMIDE

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of patients achieving successful donor engraftment

    (absolute neutrophil count > 500/uL and ≥ 90% donor cell chimerism)

    Measured: 60 Days

Secondary outcomes (1)

  1. 100-day non-relapse mortality (NRM) rate.

    The regimen will be considered as safe if 100d NRM rate is <=5%, and not safe if 100d NRM rate is ≥25%.

    Measured: 100 Days

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted16 Jul 2020
  2. Study start14 Aug 2020
  3. Primary completion22 Oct 2023
  4. Study completion19 Aug 2024

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
25 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04473911
Sponsor's protocol ID
19-336
Status last verified
Jul 2025
First submitted
13 Jul 2020
Last updated
20 Jul 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: The Dana-Farber / Harvard Cancer Center encourages and supports the responsible and ethical sharing of data from clinical trials. De-identified participant data from the final research dataset used in the published manuscript may only be shared under the terms of a Data Use Agreement. Requests may be directed to, please contact the Sponsor Investigator or designee\]. The protocol and statistical analysis plan will be made available on Clinicaltrials.gov only as required by federal regulation or as a condition of awards and agreements supporting the research.

Publications

  • DeFillipp Z, Horick NK, Gallagher KME, Lee WH, Kiana JG, Diana B, Gebretsadik R, Lee DD, Chung J, El-Jawahri A, Frigault MJ, McAfee S, Newcomb RA, O'Donnell PV, Spitzer TR, Westervelt P, Chen YB. PTCy, sirolimus, MMF and RGI-2001 to explore regulatory T-cell expansion following haploidentical peripheral blood stem cell transplantation: a pilot study. Leuk Lymphoma. 2026 Oct 20:1-9. doi: 10.1080/10428194.2026.2747545. Online ahead of print.

    PubMed 42897991

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing FLUDARABINE, CYCLOPHOSPHAMIDE and TBI in people with GVHD and AML, run by Zachariah Michael DeFilipp at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 15 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 20 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of patients achieving successful donor engraftment (time frame: 60 Days). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04473911, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Jul 2025.

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